Canadian clinical trial registry

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Information is also accessible through the patient and families tab. Family friendly summaries are created and reviewed by our advocacy partners. The information is updated to the best of our knowledge but might not reflect the latest information. Note that most studies are only available at a limited number of sites, please click on ‘further information’ for details. Studies, particularly early phase trials, may also temporarily close to enrolment or not have slots available for all treatment groups. In all cases, study teams at individual C17 centres will have the most up-to-date information.

114 results found

Title
Status

 

OPTIMISE - ARM C - AN INTERNATIONAL PILOT PHASE 2 MULTI-CENTRE STUDY OF THE EFFICACY OF OPDUALAG, A FIXED DOSE COMBINATION OF NIVOLUMAB AND RELATLIMAB, IN CHILDREN, ADOLESCENTS AND YOUNG ADULTS WITH RELAPSED AND REFRACTORY SOLID TUMOURS WITH HIGH IMMUNE INFILTRATION AND/OR REPLICATION REPAIR DEFICIENCY

Open

OPTIMISE - ARM C - AN INTERNATIONAL PILOT PHASE 2 MULTI-CENTRE STUDY OF THE EFFICACY OF OPDUALAG, A FIXED DOSE COMBINATION OF NIVOLUMAB AND RELATLIMAB, IN CHILDREN, ADOLESCENTS AND YOUNG ADULTS WITH RELAPSED AND REFRACTORY SOLID TUMOURS WITH HIGH IMMUNE INFILTRATION AND/OR REPLICATION REPAIR DEFICIENCY

Go to Health Care Provider version

DiagnosisRelapsed or refractory extra-cranial solid and CNS tumourStudy StatusOpen
PhaseI/II
Age12 years and olderRandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Opdualag (IV)
Last Posted Update2026-05-13
ClinicalTrials.gov #NCT06208657
International Sponsor
Australian & New Zealand Children's Haematology/Oncology Group
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel Morgenstern
BC Children's Hospital - Dr. Rebecca Deyell
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

 

 

Study Description

 This study is eligible for STEP-1 funding. Find more information here

 

This study is testing Opdualag, a medicine that combines two drugs, in children, teens, and young adults (12 years and older) whose cancers have returned or not responded to treatment. Some participants will have tumors that show high immune activity, while others will have tumors that did not respond to previous immunotherapy.

The study will look at how well Opdualag works, any side effects, and how treatment affects quality of life. Blood and tissue samples will also be collected to help researchers better understand the cancer and the immune system.

Inclusion Criteria
  • Patients must be 12 years or older with cancer that has come back or didn’t respond to treatment.
  • Patients have a cancer (outside or inside the brain/spine) that has come back or not responded to treatment
  • There are two groups:
    • Tumors with a strong immune response
    • Tumors with a DNA repair problem has worsened during or after prior immunotherapy
  • Patients must have measurable disease confirmed by appropriate imaging methods
  • Must meet all organ function requirements
  • Must be up and about at least 50% of waking hours (wheelchair allowed)
  • Enough time must have passed from any previous treatment; the study team will let you know the timelines
  • Patients must remain abstinent or use adequate birth control as explained by the study team

DECRYPT-BABYBRAIN - A Pilot Study of Intrathecal Topotecan and Maintenance Chemotherapy in the Post-consolidation Setting for the Treatment of High-risk Embryonal Central Nervous System Tumours in Children Less Than 6 Years of Age

Open

DECRYPT-BABYBRAIN - A Pilot Study of Intrathecal Topotecan and Maintenance Chemotherapy in the Post-consolidation Setting for the Treatment of High-risk Embryonal Central Nervous System Tumours in Children Less Than 6 Years of Age

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DiagnosisCentral nervous system (CNS) HR-EBT: Embryonal Tumor (various), Group 3 and 4 Medulloblastoma, Atypical Teratoid Rhabdoid Tumor, Pineoblastoma, CNS Neuroblastoma, MedulloepitheliomaStudy StatusOpen
PhaseI
Ageup to (and including) 6 Years oldRandomisationNO
Line of treatmentFirst line treatment
Routes of Treatment AdministrationInduction Phase: - Drug: Double Therapy (Cytarabine, Hydrocortisone) - intrathecal (IT) - Drug: Cisplatin - intravenous (IV) - Drug: Vincristine - intravenous (IV) - Drug: Etoposide - intravenous (IV) - Drug: Cyclophosphamide - intravenous (IV) - Drug: Mesna - intravenous (IV) - Drug: Filgrastim - subcutaneous or intravenous (SC or IV) Consolidation Phase: - Drug: Carboplatin - intravenous (IV) - Drug: Thiotepa - intravenous (IV) - Drug: Filgrastim - subcutaneous or intravenous (SC or IV) Maintenance Arms (A and/or B): - Drug: Topotecan - intrathecal (IT) - Drug (Maintenance A Only): Tamoxifen - oral (PO) - Drug: ISOtretinoin - oral (PO) - Drug (Maintenance B Only): Celecoxib - oral (PO) - Drug (Maintenance B Only): Temozolomide - oral (PO) - Drug (Maintenance B Only): Cyclophosphamide - oral (PO) - Drug (Maintenance B Only): Etoposide - oral (PO)
Last Posted Update2026-05-13
ClinicalTrials.gov #NCT06942039
International Sponsor
C17 Council
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Annie Huang
CHU Ste. Justine - Dr. Sébastien Perreault
Alberta Children's Hospital - Dr. Lucie Lafay-Cousin
BC Children's Hospital - Dr. George Michaiel
London Children's Hospital - Dr. Shayna Zelcer
Stollery Children's Hospital - Dr. Liana Nobre
CHU de Quebec - Dr. Valerie Larouche
CancerCare Manitoba - Dr. Divya Subburaj
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact
Dr. Alexandra Zorzi
Dr. Shayna Zelcer
 
Social worker/patient navigator contact
Cindy Milne Wren
Jessica Mackenzie Harris
 
Clinical research contact
Mariam Mikhail
Medical contact
Dr. Victor Lewis

 

Social worker/patient navigator contact
Wendy Pelletier
Clinical research contact
Debra Rich
Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

Medical contact
Dr. Sarah McKillop
Dr. Sunil Desai

 

 

Social worker/patient navigator contact
Danielle Sikora
 Michelle Woytiuk 
Jaime Hobbs
Clinical research contact
Amanda Perreault
Medical contact
Raoul Santiago
 
Social worker/patient navigator contact
Isabelle Audet
 
Clinical research contact
Barbara Desbiens
 

 

Medical contact
Dr. Magimairajan Vanan
Social worker/patient navigator contact
Rhéanne Bisson
 
Clinical research contact
Rebekah Hiebert
Megan Ridler
Kathy Hjalmarsson

 

 

 

 

Study Description

 

If you are a long-distance patient of >160 km, one way - you may be eligible for STEP-1 travel funding. Find more information here

 

A pilot study to see if it is possible and safe to add medicine given into the spinal fluid (intrathecal chemotherapy) and continued treatment (maintenance therapy) after strong chemotherapy for young children under 6 years old who have newly diagnosed high-risk brain tumors.

Inclusion Criteria
  • Age: Children 6 years old or younger at the time their brain tumor is confirmed.
  • Tumor types: Certain rare, aggressive brain or spinal tumors, including ATRT, medulloblastoma (group 3 or 4), pineoblastoma, ETMR, and other similar embryonal brain tumors.
  • MRI scans: MRI of the brain and spine (with and without contrast) must be done before and after surgery.
  • Lumbar puncture (spinal tap): A sample of spinal fluid is recommended (if safe to do) before or after surgery, but not required.
  • Must meet all lab and organ function requirements
  • Must be well enough to be up and about at least 50% of waking hours

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team. 

BCC015 - Phase II Trial of Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

Closed to enrollment

BCC015 - Phase II Trial of Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

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DiagnosisNeuroblastomaStudy StatusClosed to enrollment
PhaseII
AgeChild, Adult - (up to 31 years)RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationEtoposide: oral DFMO: oral
Last Posted Update2026-05-13
ClinicalTrials.gov #NCT04301843
International Sponsor
Giselle Sholler
Principal Investigators for Canadian Sites
Montreal Children's Hospital – Dr. Jitka Stankova
CancerCare Manitoba – Dr. Ashley Chopek
CHU Ste-Justine – Dr. Pierre Teira
CHU Sherbrooke - Dr. Josée Brossard
Alberta Children's Hospital – Dr. Melanie Finkbeiner
CHU de Quebec - Dr. Bruno Michon
Janeway Hospital – Dr. Paul Moorehead
Centres
Medical contact
Clinical Research Unit
 
Social worker/patient navigator contact
Clinical Research Unit
 
Clinical research contact
Stephanie Badour
 
Medical contact
Dr. Magimairajan Vanan
Social worker/patient navigator contact
Rhéanne Bisson
 
Clinical research contact
Rebekah Hiebert
Megan Ridler
Kathy Hjalmarsson

 

 

Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact
Dr. Victor Lewis

 

Social worker/patient navigator contact
Wendy Pelletier
Clinical research contact
Debra Rich
Medical contact
Raoul Santiago
 
Social worker/patient navigator contact
Isabelle Audet
 
Clinical research contact
Barbara Desbiens
 

 

Medical contact
Dr. Josee Brossard 
Social worker/patient navigator contact
Please Contact Site Directly
 
Clinical research contact
Please Contact Site Directly 
 
Medical contact
Dr. Paul Moorehead
 
Social worker/patient navigator contact
Stephanie Eason
 
Clinical research contact
Bev Mitchell
 

 

 

Study Description

This study is to assess the effectiveness of a drug called Difluoromethylornithine (DFMO) in combination with etoposide for patients with neuroblastoma that has come back, or is not responidng to current treatment. DFMO is a medication taken by mouth. It is an blocker of ornithine decarboxylase, an enzyme involved with polyamine biosynthesis in the cancer cells, including neuroblastoma cells. 

Inclusion Criteria
  • Patients less than 31 years of age 
  • Diagnosis of neuroblastoma that has come back or is not responding to treatment 
  • Patients can have active or no active disease at the time of study start
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

 

BCC021 - Phase I/II Study of Silmitasertib (CX-4945) in Combination With Chemotherapy in Children and Young Adults With Relapsed Refractory Solid Tumors

Open

BCC021 - Phase I/II Study of Silmitasertib (CX-4945) in Combination With Chemotherapy in Children and Young Adults With Relapsed Refractory Solid Tumors

Go to Health Care Provider version

DiagnosisRecurrent Neuroblastoma, Ewing's Sarcoma, Osteosarcoma, Rhabdomyosarcoma, LiposarcomaStudy StatusOpen
PhaseI/II
Ageup to 30 YearsRandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Silmitasertib (Oral) Other Names: CX-4945 Drug: Irinotecan (IV) Drug: Temozolomide (Oral) ± Drug: Vincristine (IV)
Last Posted Update2026-05-13
ClinicalTrials.gov #NCT06541262
International Sponsor
Milton S. Hershey Medical Center
Principal Investigators for Canadian Sites
CHU Ste-Justine - Dr. Monia Marzouki
Centres
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 

 

 

Study Description

 

The purpose of this study is to learn more about how safe and effective a drug called silmitasertib (an oral pill) is in combination with other FDA approved chemotherapy drugs to treat certain kinds of solid tumours. 

Inclusion Criteria
  • Participants must be 30 years or younger
  • A confirmed tumor type of Neuroblastoma, Ewing sarcoma, Osteosarcoma, Rhabdomyosarcoma, or Liposarcoma that has come back or not responded to standard treatment
  • Doctors must be able to see signs of cancer on a scan or in the bone marrow
  • Must have recovered from past treatments, with enough time passed since chemo, radiation, immunotherapy, or transplant
  • Must meet all organ function and bloodwork requirements
  • Must be up and about for 50% of waking hours
  • If patient can become pregnant, a negative pregnancy test and adequate birth control is required
  • Signed informed consent

AREN1921 - Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)

Open

AREN1921 - Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)

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DiagnosisAnaplastic Wilms Tumor, Recurrent Wilms TumorStudy StatusOpen
PhaseII
Ageup to 30 YearsRandomisationNO
Line of treatmentFirst line treatment, Disease relapse or progression
Routes of Treatment AdministrationChemotherapy medications, all given intravenously (Carboplatin, Cyclophosphamide, Doxorubicin, Etoposide, Ifosfamide, Irinotecan, Topotecan, Vincristine)
Last Posted Update2026-05-13
ClinicalTrials.gov #NCT04322318
International Sponsor
Children's Oncology Group
Principal Investigators for Canadian Sites
Alberta Children's Hospital - Dr. Victor Lewis
BC Children's Hospital - Dr. Rebecca Deyell
CancerCare Manitoba - Dr. Ashley Chopek
McMaster Children's Hospital - Dr. Uma Athale
Stollery Children's Hospital - Dr. Sarah McKillop
Western Children's Hospital - Dr. Shayna Zelcer
Children's Hospital of Eastern Ontario (CHEO) - Dr. Donna Johnston
Hospital for Sick Children - Dr. Daniel Morgenstern
Montreal Children's Hospital - Dr. Jitka Stankova
CHU Quebec - Dr. Bruno Michon
CHU Ste. Justine - Dr. Monia Marzouki
IWK Health Centre - Dr. Craig Erker
Janeway Hospital - Dr. Lisa Goodyear
CHU Sherbrooke - Dr. Josée Brossard
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

Medical contact
Dr. Magimairajan Vanan
Social worker/patient navigator contact
Rhéanne Bisson
 
Clinical research contact
Rebekah Hiebert
Megan Ridler
Kathy Hjalmarsson

 

 

Medical contact
Dr. Carol Portwine
 
Social worker/patient navigator contact
Jane Cassano 
 
Clinical research contact
Sabrina Millson
 
 
Medical contact
Dr. Donna Johnston
 
Dr. Lesleigh Abbott
 
Dr. Nirav Thacker
 
Social worker/patient navigator contact
Sherley Telisma
 
Clinical research contact
Doaa Abdelfattah
 
Isabelle Laforest
 
 
Medical contact
Dr. Alexandra Zorzi
Dr. Shayna Zelcer
 
Social worker/patient navigator contact
Cindy Milne Wren
Jessica Mackenzie Harris
 
Clinical research contact
Mariam Mikhail
Medical contact
Clinical Research Unit
 
Social worker/patient navigator contact
Clinical Research Unit
 
Clinical research contact
Stephanie Badour
 
Medical contact
Dr. Victor Lewis

 

Social worker/patient navigator contact
Wendy Pelletier
Clinical research contact
Debra Rich
Medical contact
Raoul Santiago
 
Social worker/patient navigator contact
Isabelle Audet
 
Clinical research contact
Barbara Desbiens
 

 

Medical contact
Dr. Sarah McKillop
Dr. Sunil Desai

 

 

Social worker/patient navigator contact
Danielle Sikora
 Michelle Woytiuk 
Jaime Hobbs
Clinical research contact
Amanda Perreault
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact
Dr. Paul Moorehead
 
Social worker/patient navigator contact
Stephanie Eason
 
Clinical research contact
Bev Mitchell
 
Medical contact
Dr. Josee Brossard 
Social worker/patient navigator contact
Please Contact Site Directly
 
Clinical research contact
Please Contact Site Directly 
 

 

 

Study Description

In this study, researchers want to find out if

  • they can improve treatment for participants with newly diagnosed diffuse anaplastic Wilms tumour (DAWT). In hopes of finding a better therapy to fight against the cancer, participants will be given chemotherapy drugs and radiation therapy in this study. The chemotherapy drugs that participants will be receiving are often given to fight against cancer and together, the treatment is known as Regimen UH-3. This study looks at how well Regimen UH-3 works when given to children and young adults with newly diagnosed DAWT.
  • they can improve treatment for participants with "standard risk" favourable histogy Wilms tumour (FWHT) that has come back (relapsed). In hopes of finding a better therapy to fight against the cancer, participants will be given chemotherapy drugs and radiation therapy in this study. The chemotherapy drugs that participants will be receiving are often given to fight against cancer and together, the treatment is known as Regimen UH-3. This study looks at how well Regimen UH-3 works when given to children and young adults with "standard risk" favourable histogy Wilms tumour (FWHT) that has come back (relapsed).
  • they can improve treatment for participants with "high-risk or very high-risk" favourable histogy Wilms tumour (FWHT) that has come back (relapsed). In hopes of finding a better therapy to fight against the cancer you have, participants will be given chemotherapy drugs and radiation therapy in this study. The chemotherapy drugs that participants will be receiving are often given to fight cancer and together, the regimen is known as Regimen ICE/Cyclo/Topo. This study looks at how well this regimen works when given to children and young adults with "high-risk or very high-risk" favourable histogy Wilms tumour (FWHT) that has come back (relapsed).

Drugs used in chemotherapy regimens work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

 

Inclusion Criteria
  • Age less than 30 years
  • New diagnosis of diffuse anaplastic Wilms tumour
  • OR a diagnosis of Wilms tumour at first relapse (tumour that has come back, and must have been "favourable histology" at initial diagnosis)
  • Patient must be capable of all self care - such that they are out of bed >50% of day
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

AHEP1531 - Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

Closed

AHEP1531 - Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

Go to Health Care Provider version

DiagnosisHepatocellular Carcinoma, Malignant Liver Neoplasm, Fibrolamellar Carcinoma, Hepatoblastoma Study StatusClosed
PhaseII/III
AgeChild, Adult - (up to 30 Years)RandomisationYES
Line of treatmentFirst line treatment
Routes of Treatment AdministrationCisplatin: IV, Other drugs are given as usually administered for hepatoblastoma/liver cancer therapy
Last Posted Update2026-05-11
ClinicalTrials.gov #NCT03533582
International Sponsor
Children's Oncology Group
Principal Investigators for Canadian Sites
Alberta Children's Hospital - Dr. Victor A. Lewis
University of Alberta Hospital (Not Affiliated with U-Link) - Dr. Sarah J. McKillop
CancerCare Manitoba - Dr. Ashley Chopek
IWK Health Centre - Dr. Craig Erker
Hamilton Health Sciences Centre, McMaster University - Dr. Uma H. Athale
Cancer Centre of Southeastern Ontario at Kingston General Hospital - Dr. Laura Wheaton
Children's Hospital of Western Ontario - Dr. Shayna M. Zelcer
The Hospital for Sick Children - Dr. Furqan Shaikh
Montreal Children's Hospital - Dr. Sharon B. Abish
CHU Ste-Justine - Dr. Yvan Samson
CHU de Quebec - Dr. Bruno Michon
Centres
Medical contact
Dr. Victor Lewis

 

Social worker/patient navigator contact
Wendy Pelletier
Clinical research contact
Debra Rich
Medical contact
Dr. Magimairajan Vanan
Social worker/patient navigator contact
Rhéanne Bisson
 
Clinical research contact
Rebekah Hiebert
Megan Ridler
Kathy Hjalmarsson

 

 

Medical contact
Dr. Craig Erker
Dr. Conrad Fernandez 
Dr. Ketan Kulkarni 
 
Social worker/patient navigator contact
Rhonda Brophy
 
Clinical research contact
Tina Bocking
 
Medical contact
Dr. Carol Portwine
 
Social worker/patient navigator contact
Jane Cassano 
 
Clinical research contact
Sabrina Millson
 
 
Medical contact
Dr. Laura Wheaton
Dr. Mariana Silva
 
Social worker/patient navigator contact
Jessica Amey
 
Clinical research contact
Heather McLean
 
Medical contact
Dr. Alexandra Zorzi
Dr. Shayna Zelcer
 
Social worker/patient navigator contact
Cindy Milne Wren
Jessica Mackenzie Harris
 
Clinical research contact
Mariam Mikhail
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Clinical Research Unit
 
Social worker/patient navigator contact
Clinical Research Unit
 
Clinical research contact
Stephanie Badour
 
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact
Raoul Santiago
 
Social worker/patient navigator contact
Isabelle Audet
 
Clinical research contact
Barbara Desbiens
 

 

 

 

Study Description

This trial studies how well cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or liver cancer after surgery. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan, sorafenib, gemcitabine and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving combination chemotherapy after surgery may kill more tumor cells.


Inclusion Criteria
  • Patients must be newly diagnosed with hepatic malignancies (liver cancers) such as hepatoblastoma or hepatocellular carcinoma
  • Patients must be under 30 years of age 
  • All patients and/or their parents or legal guardians must sign a written informed consent

ACNS1821 - A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)

Open

ACNS1821 - A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)

Go to Health Care Provider version

DiagnosisHigh-Grade Glioma (HGG) without H3 K27M mutationStudy StatusOpen
PhaseI/II
Age12 Months to 21 YearsRandomisationNO
Line of treatmentFirst line treatment
Routes of Treatment AdministrationDrug: Selinexor (oral) Radiation: Radiation therapy
Last Posted Update2026-04-28
ClinicalTrials.gov #NCT05099003
International Sponsor
National Cancer Institute (NCI)
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Julie Bennett
BC Children's Hospital - Dr. Rebecca Deyell
CancerCare Manitoba - Dr. Ashley Chopek
CHU Quebec - Dr. Bruno Michon
CHU Ste Justine - Dr. Monia Marzouki
IWK - Dr. Craig Erker
Stollery Children's Hospital - Dr. Sarah McKillop
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

Medical contact
Dr. Magimairajan Vanan
Social worker/patient navigator contact
Rhéanne Bisson
 
Clinical research contact
Rebekah Hiebert
Megan Ridler
Kathy Hjalmarsson

 

 

Medical contact
Raoul Santiago
 
Social worker/patient navigator contact
Isabelle Audet
 
Clinical research contact
Barbara Desbiens
 

 

Medical contact
Dr. Craig Erker
Dr. Conrad Fernandez 
Dr. Ketan Kulkarni 
 
Social worker/patient navigator contact
Rhonda Brophy
 
Clinical research contact
Tina Bocking
 
Medical contact
Dr. Sarah McKillop
Dr. Sunil Desai

 

 

Social worker/patient navigator contact
Danielle Sikora
 Michelle Woytiuk 
Jaime Hobbs
Clinical research contact
Amanda Perreault
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 

 

 

Study Description

 

If you are a long-distance patient of >160 km, one way - you may be eligible for STEP-1 travel funding. Find more information here

 

This clinical trial is testing a new treatment for children and young adults with aggressive brain tumors, including DIPG and high grade glioma with a specific genetic change (H3 K27M mutation). The treatment combines standard radiation therapy with a new medicine called selinexor, which may help stop cancer cells from growing and shrink tumors. Patients receive radiation for 5-7 weeks and take selinexor pills weekly during and after radiation for up to two years. The study will first find the safest dose of selinexor and then test how well it works. MRIs and follow-up visits help monitor progress. Researchers hope this treatment will improve outcomes for these difficult-to-treat brain tumors.

NOTE: Enrollment for DIPG and DMG has been discontinued. This study is only opened for patients with HGG without a H3 K27M mutation. 

Inclusion Criteria

Pre-Enrollment

  1. Age: Patients must be 25 years old or younger.
  2. Diagnosis: Must have a newly diagnosed high-grade glioma (HGG) that hasn’t spread.
  3. Consent: A parent or guardian, or the patient, must sign a consent form.
  4. Samples (non-DIPG tumours): Tumour samples from surgery or biopsy need to be submitted soon after the procedure (ideally within 5 days).

Main Enrollment

  1. Age: Patients must be between 1 and 21 years old.
  2. Diagnosis:
    • DIPG: Must meet specific imaging or biopsy criteria. CLOSED
    • HGG: Must be newly diagnosed, without certain genetic mutations. OPEN
  3. Patients need to be in good overall health with normal lab results and no severe symptoms.
  4. Enrollment must happen within 31 days of diagnosis or surgery.
  5. A signed consent form is required.

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team. 

VICTORY (OZM-138) - VICTORY: A Pilot Study to Investigate Safety and Efficacy of Weekly Combination of Intravenous Vinblastine With Oral Type II RAF Inhibitor Tovorafenib in Pediatric Patients With Recurrent/Progressive RAF Altered Low Grade Gliomas

Open

VICTORY (OZM-138) - VICTORY: A Pilot Study to Investigate Safety and Efficacy of Weekly Combination of Intravenous Vinblastine With Oral Type II RAF Inhibitor Tovorafenib in Pediatric Patients With Recurrent/Progressive RAF Altered Low Grade Gliomas

Go to Health Care Provider version

DiagnosisLow-grade GliomaStudy StatusOpen
PhaseI
AgeUp to 25 YearsRandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Tovorafenib oral (immediate-release tablets or powder for reconstitution) Drug: Vinblastine IV
Last Posted Update2026-04-28
ClinicalTrials.gov #NCT06381570
International Sponsor
IIT - The Hospital for Sick Children
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Uri Tabori
HHSC/McMaster - Dr. Adam Fleming
CHEO - Dr. Nirav Thacker
Stollery Children's Hospital - Dr. Liana Nobre
BC Children's Hospital - Dr. Sylvia Cheng
CHU Ste Justine - Dr. Sébastien Perreault
Alberta Children's Hospital - Dr. Lucie Lafay-Cousin
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Dr. Carol Portwine
 
Social worker/patient navigator contact
Jane Cassano 
 
Clinical research contact
Sabrina Millson
 
 
Medical contact
Dr. Donna Johnston
 
Dr. Lesleigh Abbott
 
Dr. Nirav Thacker
 
Social worker/patient navigator contact
Sherley Telisma
 
Clinical research contact
Doaa Abdelfattah
 
Isabelle Laforest
 
 
Medical contact
Dr. Sarah McKillop
Dr. Sunil Desai

 

 

Social worker/patient navigator contact
Danielle Sikora
 Michelle Woytiuk 
Jaime Hobbs
Clinical research contact
Amanda Perreault
Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact
Dr. Victor Lewis

 

Social worker/patient navigator contact
Wendy Pelletier
Clinical research contact
Debra Rich

 

 

Study Description

If you are a long-distance patient of >160 km, one way - you may be eligible for STEP-1 travel funding. Find more information here

 

VICTORY is an early phase study for patients under 25 years who have a type of brain tumor called low grade glioma (LGG) that coms back or gets worse (recurrent or progressive). To participate on this study, the LGG must have certain genetic changes called CRAF or BRAF alterations.

The study has two parts:

Phase A: We are figuring out the best dose of a combination of two medicines, vinblastine and tovorafenib. Patients will take these medicines for a certain number of months, then just tovorafenib for a few more months. Their progress will be checked regularly with scans.

Phase B: After we find the right dose in Phase A, we'll test how well the combination of these drugs works. Patients will take these medicines for a certain number of months, then just tovorafenib for a few more months. Their progress will be checked regularly with scans.

Patients will be in the study for about two years unless their tumor gets worse (disease progression), they have bad side effects or they decide to withdrawal from the study.

Inclusion Criteria
  • Patients must be less than or equal to 25 years of age at the time of enrollment
  • Patient and/or their parent/guardian must sign an informed consent form to be enrolled on study
  • Patients must have a diagnosis of low grade glioma (LGG) with a genetic alteration called BRAF or CRAF alteration that has come back (recurrent) or not responded to previous treatment (progressive)
  • Patients must have completed at least 1 other therapy prior to enrollment on this study and recovered from its effects.
  • Patients must have adequate performance status (daily activities which they are able to do) 
  • Must have adequate organ function in bone marrow, kidneys, liver, heart and thyroid
  • Willingness of male and female patients with reproductive potential to use double effective birth control methods, defined as one used by the patient and another by his/her partner, for the duration of treatment and for 180 days following the last dose of study drug. 
  • Ability to swallow tablets or liquid, or gastric access via a nasal or gastric tube.

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team.